Validation of childhood lupus specific targets: ensuring accurate assessment of disease control in younger, lighter paediatric patients.

Objectives: To validate novel childhood-onset systemic lupus erythematosus (cSLE) T2T targets including Childhood Lupus Low Disease Activity State (cLLDAS), cSLE Clinical Remission on steroids (cCR), and cSLE Clinical Remission off steroids (cCR-0), as compared with adult-onset SLE (aSLE) targets.

Methods: Attainment of the aforementioned cSLE-specific and aSLE-specific targets (LLDAS, DORIS 2021 Remission) was assessed at each visit, in UK JSLE Cohort Study patients. Univariable and multivariable Prentice-Williams-Peterson (PWP) gap-time models investigated the impact of target attainment on new damage and severe flare.

Contributors to Organ Damage in Childhood Lupus: Corticosteroid Use and Disease Activity.

Background: Awareness of paediatric-specific predictors of damage in Childhood-lupus is needed to inform mitigation measures.

Objectives: To ascertain how clinical and demographic variables correlate with damage accrual and identify predictors of damage.

Methods: Analysis included UK JSLE Cohort Study participants.

Panel sequencing links rare, likely damaging gene variants with distinct clinical phenotypes and outcomes in juvenile-onset SLE.

Objectives: Juvenile-onset systemic lupus erythematosus (jSLE) affects 15-20% of lupus patients. Clinical heterogeneity between racial groups, age groups and individual patients suggests variable pathophysiology. This study aimed to identify highly penetrant damaging mutations in genes associated with SLE/SLE-like disease in a large national cohort (UK JSLE Cohort Study) and compare demographic, clinical and laboratory features in patient sub-cohorts with 'genetic' SLE vs remaining SLE patients.

Evaluation of Serious Infection in Pediatric Patients with Low Immunoglobulin Levels Receiving Rituximab for Granulomatosis with Polyangiitis or Microscopic Polyangiitis.

Introduction: The aim of this work was to assess the impact of prolonged low immunoglobulin (IgG or IgM) serum concentrations on the potential cumulative serious infection (SI) risk in pediatric patients following rituximab treatment for granulomatosis with polyangiitis or microscopic polyangiitis (GPA/MPA) in PePRS.

Methods: Patients aged ≥ 2 to < 18 years received four weekly intravenous rituximab infusions of 375 mg/m and concomitant glucocorticoid taper. After 6 months, patients could receive further rituximab and/or other immunosuppressants per investigator discretion.

Attainment of low disease activity and remission targets reduces the risk of severe flare and new damage in childhood lupus.

Objectives: To assess the achievability and effect of attaining low disease activity (LDA) or remission in childhood-onset SLE (cSLE).

Methods: Attainment of three adult-SLE derived definitions of LDA (LLDAS, LA, Toronto-LDA), and four definitions of remission (clinical-SLEDAI-defined remission on/off treatment, pBILAG-defined remission on/off treatment) was assessed in UK JSLE Cohort Study patients longitudinally. Prentice-Williams-Petersen gap recurrent event models assessed the impact of LDA/remission attainment on severe flare/new damage.

Neuropsychiatric involvement in juvenile-onset systemic lupus erythematosus: Data from the UK Juvenile-onset systemic lupus erythematosus cohort study.

Introduction: Juvenile-onset systemic lupus erythematosus (JSLE) is a rare autoimmune/inflammatory disease with significant morbidity and mortality. Neuropsychiatric (NP) involvement is a severe complication, encompassing a heterogeneous range of neurological and psychiatric manifestations.

Methods: Demographic, clinical, and laboratory features of NP-SLE were assessed in participants of the UK JSLE Cohort Study, and compared to patients in the same cohort without NP manifestations.

Phase IIa Global Study Evaluating Rituximab for the Treatment of Pediatric Patients With Granulomatosis With Polyangiitis or Microscopic Polyangiitis.

Objective: To assess the safety, tolerability, pharmacokinetics, and efficacy of rituximab (RTX) in pediatric patients with granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA).

Methods: The Pediatric Polyangiitis Rituximab Study was a phase IIa, international, open-label, single-arm study. During the initial 6-month remission-induction phase, patients received intravenous infusions of RTX (375 mg/m body surface area) and glucocorticoids once per week for 4 weeks.

Limited sensitivity and specificity of the ACR/EULAR-2019 classification criteria for SLE in JSLE?-observations from the UK JSLE Cohort Study.

Objectives: This study aimed to test the performance of the new ACR and EULAR criteria, that include ANA positivity as entry criterion, in JSLE.

Methods: Performance of the ACR/EULAR-2019 criteria were compared with Systemic Lupus International Collaborating Clinics (SLICC-2012), using data from children and young people (CYP) in the UK JSLE Cohort Study (n = 482), with the ACR-1997 criteria used as reference standard. An unselected cohort of CYP positive for ANA (n = 129) was used to calculate positive/negative predictive values of the criteria.

'It is good to have a target in mind': qualitative views of patients and parents informing a treat to target clinical trial in juvenile-onset systemic lupus erythematosus.

Objective: We sought to explore patient and parental views on treatment targets, outcome measures and study designs being considered for a future JSLE treat-to-target (T2T) study.

Methods: We conducted topic-guided, semistructured interviews with JSLE patients and parents and analysed the audio recorded interviews using thematic approaches.

Results: Patients and parents differed regarding symptoms they felt would be tolerable, representing 'low disease activity'.

Clinical and laboratory phenotypes in juvenile-onset Systemic Lupus Erythematosus across ethnicities in the UK.

Systemic lupus erythematosus (SLE) is a systemic autoimmune/inflammatory disease. Patients diagnosed with juvenile-onset SLE (jSLE), when compared to individuals with adult-onset SLE, develop more severe organ involvement, increased disease activity and greater tissue and organ damage. In adult-onset SLE, clinical characteristics, pathomechanisms, disease progression and outcomes do not only vary between individuals and age groups, but also ethnicities.

The prevention and treatment of glucocorticoid-induced osteopaenia in juvenile rheumatic disease: A randomised double-blind controlled trial.

Background: Children and young people (CYP) with chronic rheumatic conditions; Juvenile Idiopathic Arthritis, Juvenile Systemic Lupus Erythematosus, Juvenile Dermatomyositis and Juvenile Vasculitis, treated with steroids, have low bone density, increased fracture risk and are likely to have suboptimal peak bone mass. There is currently no evidence base for the management of steroid-induced bone loss in children with rheumatic diseases.

Methods: We undertook a multi-centre double dummy double-blind randomised placebo controlled trial to investigate whether the bisphosphonate risedronate was superior to alfacalcidol or calcium and vitamin D supplementation in the prevention and treatment of steroid-induced osteopaenia in these children.

Multi-centre national audit of juvenile localised scleroderma: describing current UK practice in disease assessment and management.

Objective: To describe current United Kingdom practice in assessment and management of patients with juvenile localised scleroderma (JLS) compared to Paediatric Rheumatology European Society (PRES) scleroderma working party recommendations.

Methods: Patients were included if they were diagnosed with JLS and were under the care of paediatric rheumatology between 04/2015-04/2016. Retrospective data was collected in eleven UK centres using a standardised proforma and collated centrally.

Anti-inflammatory drugs and analgesics for managing symptoms in people with cystic fibrosis-related arthritis.

Background: Arthritis remains a relatively infrequent complication of cystic fibrosis, but is a cause of significant morbidity when it does occur. Two distinct types of arthritis are described in cystic fibrosis: cystic fibrosis-related arthropathy (CFA) and hypertrophic pulmonary osteoarthropathy (HPO). Management of arthritis in people with cystic fibrosis is uncertain and complex because of the underlying disease and its intense treatment.

Mucocutaneous manifestations in a UK national cohort of juvenile-onset systemic lupus erythematosus patients.

Objective: To determine whether mucocutaneous manifestations are associated with major organ involvement in a UK national cohort of juvenile-onset SLE (JSLE) patients.

Methods: JSLE patients (n = 241) from 15 different centres whose diagnosis fulfilled four or more of the ACR criteria were divided into two groups: those with at least one ACR mucocutaneous criterion (ACR skin feature positive) and those without (ACR skin feature negative) at diagnosis. The relative frequency of skin involvement was described by the paediatric adaptation of the 2004 British Isles Lupus Assessment Group (pBILAG-2004) index.

Onset of juvenile dermatomyositis following varicella infection in a 12-month-old child: a case report.

Introduction: Infections can act as a trigger for juvenile dermatomyositis, with a predominance of respiratory tract infections reported previously. We present the first case of juvenile dermatomyositis following varicella infection to be described in the literature.

Case Presentation: A 15-month-old Caucasian girl was diagnosed with juvenile dermatomyositis 3 months after a varicella infection.

Disease modifying anti-rheumatic drugs in people with cystic fibrosis-related arthritis.

Background: Arthritis remains a relatively infrequent complication of cystic fibrosis, but is a cause of significant morbidity when it does occur. Two distinct types of arthritis are described in cystic fibrosis: cystic fibrosis-related arthropathy and hypertrophic osteoarthropathy. Management of arthritis in people with cystic fibrosis is uncertain and complex because of the underlying disease and its treatment.

Anti-inflammatory drugs and analgesics for managing symptoms in people with cystic fibrosis-related arthritis.

Background: Arthritis remains a relatively infrequent complication of cystic fibrosis, but is a cause of significant morbidity when it does occur. Two distinct types of arthritis are described in cystic fibrosis: cystic fibrosis-related arthropathy (CFA) and hypertrophic pulmonary osteoarthropathy (HPO). Management of arthritis in people with cystic fibrosis is uncertain and complex because of the underlying disease and its intense treatment.

Access to care for children and young people diagnosed with localized scleroderma or juvenile SSc in the UK.

Objectives: To describe pathways of care and referral to paediatric rheumatology from onset of first symptom (noticed by the patient or their family) to diagnosis for children and young people diagnosed with localized scleroderma (LS) or juvenile SSc (jSSc).

Methods: Retrospective case note audit of patients under paediatric rheumatology care who presented during January 2005-January 2010. Data included disease subtype, sex, age at key points in the referral pathway and health care professional (HCP) contact.

Disease activity, severity, and damage in the UK Juvenile-Onset Systemic Lupus Erythematosus Cohort.

Objective: The UK Juvenile-Onset Systemic Lupus Erythematosus (JSLE) Cohort Study is a multicenter collaborative network established with the aim of improving the understanding of juvenile SLE. The present study was undertaken to describe the clinical manifestations and disease course in patients with juvenile SLE from this large, national inception cohort.

Methods: Detailed data on clinical phenotype were collected at baseline and at regular clinic reviews and annual followup assessments in 232 patients from 14 centers across the UK over 4.

British isles survey of methotrexate monitoring practice during treatment of juvenile idiopathic arthritis.

Objectives: We surveyed current pediatric rheumatology monitoring practice in methotrexate treatment of juvenile idiopathic arthritis in the British Isles, and experiences of significant side effects during methotrexate monitoring.

Methods: Single-center responses were sought from the current British Society for Pediatric and Adolescent Rheumatology membership, using a web-based survey tool.

Results: Thirty-three centers across the British Isles responded.

Disease modifying anti-rheumatic drugs in people with cystic fibrosis-related arthritis.

Background: Arthritis remains a relatively infrequent complication of cystic fibrosis, but is a cause of significant morbidity when it does occur. Two distinct types of arthritis are described in cystic fibrosis: cystic fibrosis-related arthropathy and hypertrophic osteoarthropathy. Management of arthritis in people with cystic fibrosis is uncertain and complex because of the underlying disease and its treatment.

General paediatricians and the case of resolving peanut allergy.

Children with peanut allergy are almost always advised to avoid nuts for life. There have been recent reports from academic centres that in some cases the allergy might resolve and thus these dietary restrictions can be lifted. To evaluate resolution of peanut allergy in a selected group of children in a general paediatric setting.